The myelodysplastic syndromes: current approaches to therapy

To review the current therapeutic options for patients with the myelodysplastic syndromes.

Studies reported between 1968 and September 1989 were identified through computer searches using MEDLINE and through extensive searching of bibliographies of identified articles.

All articles, abstracts, and reviews were evaluated, and those that contained therapeutic data were analyzed for response rates, survival, and toxicity.

Therapies for the myelodysplastic syndromes have included hormones, chemotherapy, bone marrow transplantation, and differentiating agents, including hematopoietic growth factors. With the exception of bone marrow transplantation, none is curative or increases survival. Hematopoietic growth factors are of interest for clinical trials, because they increase the number of neutrophils and, occasionally, the number of platelets in patients with the myelodysplastic syndromes. Nonetheless, hematopoietic growth factors are not without toxicity; most notably, they are associated with a risk for acceleration to acute myeloid leukemia, and their effect on survival remains unknown.

Standard therapy for the myelodysplastic syndromes is supportive care. When the disease progresses, patients with aggressive histologic or other poor-risk features should be considered for aggressive chemotherapy, with or without growth factors, or bone marrow transplantation. Patients with good prognostic features are candidates for therapy with growth factors or other potential differentiating agents. Carefully designed and conducted clinical trials that incorporate correlative laboratory science are essential to developing a more rational approach to therapy.